Mitochondrial augmentation of hematopoietic stem cells in children with single large-scale mitochondrial DNA deletion syndromes
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DOI: 10.1126/scitranslmed.abo3724
Abstract: Patients with single large-scale mitochondrial DNA (mtDNA) deletion syndromes (SLSMDs) usually present with multisystemic disease, either as Pearson syndrome in early childhood or as Kearns-Sayre syndrome later in life. No disease-modifying therapies exist for SLSMDs.… read more here.
Keywords: mitochondrial augmentation; dna; deletion syndromes; mitochondrial dna ... See more keywords
T-Lymphoblastic Lymphoma in a Patient with Pearson Syndrome Following Mitochondrial Augmentation
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DOI: 10.1182/blood-2024-199972
Abstract: Pearson syndrome (PS) is an ultra-rare childhood disease caused by a deletion in the mitochondrial DNA (mtDNA). PS is associated with bone marrow dysfunction at young age, with later progression to multisystem dysfunction. A few… read more here.
Keywords: age; mitochondrial augmentation; pearson syndrome; tumor ... See more keywords
Mitochondrial Augmentation of Hematopoietic Stem and Progenitor Cells As a New Therapeutic Approach for Myelodysplasia
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DOI: 10.1182/blood-2024-200129
Abstract: Currently the only curative treatment option for patients with myelodysplastic syndromes (MDS) is allogeneic bone marrow transplant. However, allogeneic transplant is not feasible for the majority of MDS patients. Several studies have identified that mitochondrial… read more here.
Keywords: augmentation; mds; mitochondrial augmentation; bone marrow ... See more keywords